Autophagy in Gaucher disease due to saposin C deficiency

Autophagy. 2011 Jan;7(1):94-5. doi: 10.4161/auto.7.1.13884. Epub 2011 Jan 1.

Abstract

Gaucher disease, due to a deficit of glucosylceramidase or, rarely, of its activator saposin C, is characterized by accumulation of glucosylceramide in the lysosomes of monocyte/macrophage lineage. In our study we demonstrate that saposin C deficiency due to mutations involving a cysteine residue results in increased autophagy. Autophagy was monitored by LC3 analysis and confirmed by electron microscopy; we observed a correlation among saposin C mutation, Gaucher phenotype and increased autophagy.

MeSH terms

  • Autophagy*
  • Fibroblasts / metabolism
  • Fibroblasts / pathology
  • Gaucher Disease / genetics
  • Gaucher Disease / pathology*
  • Humans
  • Mutation / genetics
  • Saposins / deficiency*
  • Saposins / genetics
  • Saposins / metabolism

Substances

  • Saposins